Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome
Key Secondary Objective
• To investigate the efficacy of treatment with oral Bionetide versus placebo on ability to communicate in girls and women with Rett syndrome
Female subjects 5 to 20 years of age, inclusive, at Screening
Body weight ≥12 kg at Screening
Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
Has classic/typical Rett syndrome (RTT)
Has been treated with insulin within 12 weeks of Baseline
Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
Has a history of, or current, cerebrovascular disease or brain trauma
Has significant, uncorrected visual or uncorrected hearing impairment
Biomed Industries, Inc.
Lead sponsor
Bioneurals Ltd
Collaborator